Outcome
Type |
Measure |
Description |
Time frame |
Safety issue |
Primary |
Number of Patients With Adverse Events (AEs), Serious Adverse Events (SAEs) and Deaths as a Measure of Safety and Tolerability |
Safety was monitored throughout the study. |
After 78 days in Part A and after 85 days in Part B. |
|
Primary |
Number of Mild, Moderate and Severe Adverse Events as a Measure of Safety and Tolerability |
Safety was monitored throughout the study. |
After 78 days in Part A and after 85 days in Part B. |
|
Primary |
Mean Percent Change From Baseline in Thigh Muscle Volume in Part B, Cohort 5 |
Thigh muscle volume was assessed by magnetic resonance imaging (MRI). Change from baseline was calculated from the ratio of the post-baseline mean value to the baseline mean value: [(Day 85/baseline) - 1)] x 100. A positive change from baseline indicates improvement. |
Baseline, Day 85 |
|
Secondary |
Mean Change From Baseline in Score on the Adult Myopathy Assessment Tool (AMAT) in Part B, Cohort 5 |
The AMAT rated physical function and muscle endurance, with higher scores indicating better performance. The tool includes 7 timed functional tasks rated on a scale from 0 - 21 and 6 endurance tasks rated on a scale from 0 - 24. The range for the total score was from 0 (worst) to 45 (best). A positive change from baseline indicates improvement. |
Baseline, Day 85 |
|
Secondary |
Mean Change From Baseline in Total Lean Body Mass (LBM) in Part B, Cohort 5 |
LBM was assessed by dual-energy X-ray (DXA) absorptiometry. A positive change from baseline indicate improvement. |
Baseline, Day 85 |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 1 |
Serum samples were obtained for PK assessment. |
Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part A, Cohort 2 |
Serum samples were obtained for PK assessment. |
Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part A, Cohort 1 |
Serum samples were obtained for PK assessment. |
Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part A, Cohort 2 |
Serum samples were obtained for PK assessment. |
Day 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part A, Cohort 1 |
Serum samples were obtained for PK assessment. |
Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part A, Cohort 2 |
Serum samples were obtained for PK assessment. |
Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part A, Cohort 1 |
Serum samples were obtained for PK assessment. |
Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part A, Cohort 2 |
Serum samples were obtained for PK assessment. |
Days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part B, Cohort 4 |
Serum samples were obtained for PK assessment. |
Days 1: pre-dose, 1, 4, 24, 48 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: Observed Maximum Concentration Following Drug Administration (Cmax) in Part B, Cohort 5 |
Serum samples were obtained for PK assessment. |
Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part B, Cohort 4 |
Serum samples were obtained for PK assessment. |
Days 1: pre-dose, 1, 4, 24, 48 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: Time to Reach the Maximum Concentration After Drug Administration (Tmax) in Part B, Cohort 5 |
Serum samples were obtained for PK assessment. |
Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the Time of the Last Quantifiable Concentration (AUClast) in Part B, Cohort 5 |
Serum samples were obtained for PK assessment. |
Day 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857:The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part B, Cohort 4 |
Serum samples were obtained for the PK assessment. |
Days 1: pre-dose, 1, 4, 24, 48 hours post-dose |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857:The Area Under the Plasma Concentration-time Curve From Zero to 48 Hours (AUC0_48h) in Part B, Cohort 5 |
|
Days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to the End of the Dosing Interval Tau (AUCtau) |
Serum samples were obtained for PK assessment. |
Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: The Terminal Elimination Half-life (T1/2) |
Serum samples were obtained for PK assessment. |
Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. |
|
Secondary |
Plasma Pharmacokinetics (PK) of BVS857: The Area Under the Serum Concentration-time Curve From Time Zero to Infinity (AUCinf) |
Serum samples were obtained for PK assessment. |
Part A: days 1, 15, 29, 43: pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. Day 57: pre-dose, 1, 4, 12, 24, 48, 168, 504 hours post-dose. Part B: days 1 and 36: pre-dose, 1, 4, 24, 48 hours post-dose. Day 78: pre-dose, 1, 4, 24, 48, 168 hours post-dose. |
|
Secondary |
Compare Dose Normalized Log-transformed AUCinf Following IV and SC Administrations |
Serum samples were obtained for PK assessment. |
In Part A: days 1 and 15, pre-dose, 1, 4, 12, 24, 48, 168 hours post-dose. |
|